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Cost-Effectiveness and Heterogeneity: Using Finite Mixtures of Disease Activity Models to Identify and Analyze Phenotypes

Borg, Sixten LU ; Gerdtham, Ulf LU orcid ; Rydén, Tobias ; Munkholm, Pia ; Odes, Selwyn ; Moum, Bjørn ; Stockbrügger, Reinhold and Lindgren, Stefan LU (2015) In Working Paper / Department of Economics, School of Economics and Management, Lund University
Abstract
Heterogeneity in patient populations is an important issue in health economic evaluations, as the cost-effectiveness of an intervention can vary between patient subgroups, and an intervention which is not cost-effective in the overall population may be cost-effective in particular subgroups. Identifying such subgroups is of interest in the allocation of healthcare resources. Our aim was to develop a method for cost-effectiveness analysis in heterogeneous chronic diseases, by identifying subgroups (phenotypes) directly relevant to the cost-effectiveness of an intervention, and by enabling cost-effectiveness analyses of the intervention in each of these phenotypes. We identified phenotypes based on healthcare resource utilization, using... (More)
Heterogeneity in patient populations is an important issue in health economic evaluations, as the cost-effectiveness of an intervention can vary between patient subgroups, and an intervention which is not cost-effective in the overall population may be cost-effective in particular subgroups. Identifying such subgroups is of interest in the allocation of healthcare resources. Our aim was to develop a method for cost-effectiveness analysis in heterogeneous chronic diseases, by identifying subgroups (phenotypes) directly relevant to the cost-effectiveness of an intervention, and by enabling cost-effectiveness analyses of the intervention in each of these phenotypes. We identified phenotypes based on healthcare resource utilization, using finite mixtures of underlying disease activity models: first, an explicit disease activity model, and secondly, a model of aggregated disease activity. They differed with regards to time-dependence, level of detail, and what interventions they could evaluate. We used them for cost-effectiveness analyses of two hypothetical interventions. Allowing for different phenotypes improved model fit, and was a key step towards dealing with heterogeneity. The cost-effectiveness of the interventions varied substantially between phenotypes. Using underlying disease activity models for identifying phenotypes as well as cost-effectiveness analysis appears both feasible and useful in that they guide the decision to introduce an intervention. (Less)
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author
; ; ; ; ; ; and
organization
publishing date
type
Working paper/Preprint
publication status
published
subject
keywords
Cost-effectiveness, Disease heterogeneity, Phenotypes, Latent classes, Disease activity model, Crohn's disease
in
Working Paper / Department of Economics, School of Economics and Management, Lund University
issue
5
pages
25 pages
publisher
Department of Economics, Lund University
project
Disease activity models of Inflammatory Bowel Diseases
language
English
LU publication?
yes
id
35786f95-426f-4019-bf87-98736c5008eb (old id 5049210)
alternative location
http://swopec.hhs.se/lunewp/abs/lunewp2015_005.htm
date added to LUP
2016-04-04 10:40:01
date last changed
2019-03-08 02:54:05
@misc{35786f95-426f-4019-bf87-98736c5008eb,
  abstract     = {{Heterogeneity in patient populations is an important issue in health economic evaluations, as the cost-effectiveness of an intervention can vary between patient subgroups, and an intervention which is not cost-effective in the overall population may be cost-effective in particular subgroups. Identifying such subgroups is of interest in the allocation of healthcare resources. Our aim was to develop a method for cost-effectiveness analysis in heterogeneous chronic diseases, by identifying subgroups (phenotypes) directly relevant to the cost-effectiveness of an intervention, and by enabling cost-effectiveness analyses of the intervention in each of these phenotypes. We identified phenotypes based on healthcare resource utilization, using finite mixtures of underlying disease activity models: first, an explicit disease activity model, and secondly, a model of aggregated disease activity. They differed with regards to time-dependence, level of detail, and what interventions they could evaluate. We used them for cost-effectiveness analyses of two hypothetical interventions. Allowing for different phenotypes improved model fit, and was a key step towards dealing with heterogeneity. The cost-effectiveness of the interventions varied substantially between phenotypes. Using underlying disease activity models for identifying phenotypes as well as cost-effectiveness analysis appears both feasible and useful in that they guide the decision to introduce an intervention.}},
  author       = {{Borg, Sixten and Gerdtham, Ulf and Rydén, Tobias and Munkholm, Pia and Odes, Selwyn and Moum, Bjørn and Stockbrügger, Reinhold and Lindgren, Stefan}},
  keywords     = {{Cost-effectiveness; Disease heterogeneity; Phenotypes; Latent classes; Disease activity model; Crohn's disease}},
  language     = {{eng}},
  note         = {{Working Paper}},
  number       = {{5}},
  publisher    = {{Department of Economics, Lund University}},
  series       = {{Working Paper / Department of Economics, School of Economics and Management, Lund University}},
  title        = {{Cost-Effectiveness and Heterogeneity: Using Finite Mixtures of Disease Activity Models to Identify and Analyze Phenotypes}},
  url          = {{http://swopec.hhs.se/lunewp/abs/lunewp2015_005.htm}},
  year         = {{2015}},
}